Functional Fluidics, Detroit, MI, USA.
Division of Pediatric Critical Care Medicine, Children's Hospital of Michigan, Detroit, MI, USA.
Br J Haematol. 2021 Sep;194(6):1074-1082. doi: 10.1111/bjh.17643. Epub 2021 Sep 1.
Blood cell adhesion to P-selectin and vascular cell adhesion molecule-1 (VCAM-1) contributes to the pathophysiology of vaso-occlusion crisis (VOC) events in individuals with sickle cell disease (SCD). We evaluated the use of standardized flow adhesion biomarkers in a six-month, 35-subjects longitudinal study (ELIPSIS). Flow adhesion of whole blood on P-selectin (FA-WB-Psel) and VCAM1 (FA-WB-VCAM), and of isolated white blood cells on P-selectin (FA-WBC-Psel) and VCAM-1 (FA-WBC-VCAM) were elevated on VOC days compared with non-VOC days, but only FA-WB-Psel reached statistical significance (P = 0·015). Optimal cut-off values were established with Cox regression models for FA-WB-Psel [46 cells/mm²; hazard ratio (HR): 2·3; 95% confidence interval (CI):1·4-4·0; P = 0·01] and FA-WB-VCAM (408 cells/mm², HR:1·8; 95% CI: 0·9-3·45; P = 0·01). A combined (FA-WB-Psel and FA-WB-VCAM) multimarker risk score was also significantly (P = 0·0006) correlated with VOC risk that was two-fold higher for intermediate and 5·64-fold higher for high score. The concordance (C)-index for the multimarker score was 0·63 in the six-month period (95% CI: 0·56-0·70), indicating a better ability to distinguish patient risk of VOC, compared to individual biomarkers FA-WB-VCAM (C-index: 0·57; 95% CI: 0·49-0·65) or FA-WB-Psel (C-index: 0·58; 95% CI: 0·53-0·62). The presented multimarker score can be used to risk-stratify individuals with SCD during their steady state into low, intermediate, and high-risk strata for self-reported VOCs. Such risk stratification could help focus healthcare resources more efficiently to maintiain health, personalize treatment selection to each patient's individual needs, and potentially reduce healthcare costs.
血细胞与 P 选择素和血管细胞黏附分子-1(VCAM-1)的黏附导致镰状细胞病(SCD)患者血管闭塞危象(VOC)事件的病理生理学发生。我们在一项为期 6 个月、35 例受试者的纵向研究(ELIPSIS)中评估了标准化的血流黏附生物标志物的应用。与非 VOC 日相比,VOC 日全血对 P 选择素(FA-WB-Psel)和 VCAM1(FA-WB-VCAM)的黏附以及分离的白细胞对 P 选择素(FA-WBC-Psel)和 VCAM-1(FA-WBC-VCAM)的黏附均升高,但只有 FA-WB-Psel 达到统计学意义(P=0·015)。采用 Cox 回归模型建立了 FA-WB-Psel [46 个细胞/mm²;风险比(HR):2·3;95%置信区间(CI):1·4-4·0;P=0·01]和 FA-WB-VCAM(408 个细胞/mm²,HR:1·8;95%CI:0·9-3·45;P=0·01)的最佳截断值。联合(FA-WB-Psel 和 FA-WB-VCAM)多标志物风险评分也与 VOC 风险显著相关(P=0·0006),中危评分患者的风险增加 2 倍,高危评分患者的风险增加 5.64 倍。6 个月时多标志物评分的一致性(C)指数为 0·63(95%CI:0·56-0·70),表明与单独的 FA-WB-VCAM(C 指数:0·57;95%CI:0·49-0·65)或 FA-WB-Psel(C 指数:0·58;95%CI:0·53-0·62)标志物相比,该评分具有更好的区分 VOC 风险的能力。提出的多标志物评分可用于在稳定状态下将 SCD 个体分层为低危、中危和高危,以报告 VOC。这种风险分层可以帮助更有效地集中医疗资源以维持健康,根据每个患者的个体需求选择个性化治疗,并有可能降低医疗保健成本。